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Pharmacyclics, J&J Win FDA Approval for Big Blood Cancer Drug

[Updated: 11:15 am PT] Investors have been projecting billions in future sales for a new blood cancer treatment from Sunnyvale, CA-based Pharmacyclics (NASDAQ: PCYC) and Johnson & Johnson, and today the two companies got the official green light from the FDA to start selling the drug.

The FDA said today it has cleared ibrutinib (Imbruvica) as a new treatment for patients with mantle cell lymphoma, which affects about 6 percent of the patients classified as having non-Hodgkin’s lymphoma. The FDA gave this drug one of its new “breakthrough” designations, and treated it as such, by approving the drug for sale more than three months ahead of its legal review deadline of Feb. 28.

The new drug has stirred excitement because of its ability to bind with a molecular target known as Bruton’s tyrosine kinase (BTK). This target has eluded medicinal chemists for years, but it has long been considered an important player in many kinds of malignancies. While the initial approval of the drug is limited to a small patient population, Pharmacyclics and J&J are testing it against a wide range of cancers that involve excess proliferation of immune system B cells, including chronic lymphocytic leukemia, diffuse large B cell lymphoma, and multiple myeloma. About 3,000 patients are diagnosed each year with mantle cell lymphoma, and about 11,000 people in the U.S. have the disease, according to IMS Health data cited by Pharmacyclics today.

The drug could end up generating $2.1 billion in sales by 2020, mostly from chronic lymphocytic leukemia, according to EP Vantage. JP Morgan analyst Cory Kasimov, in a note to clients Sept. 25, said “very few drugs come along that offer the potential for $6B+ in peak sales, but we believe ibrutinib is one of them.” [Updated to add JP Morgan quote.]

The drug is clearly a long, long way from that goal, and will need approvals in multiple patients populations to reach that goal. The FDA is still reviewing a separate application by Pharmacyclics and J&J to market the drug for chronic lymphocytic leukemia.

“Imbruvica’s approval demonstrates the FDA’s commitment to making treatments available to patients with rare diseases,” said Richard Pazdur, the director of the FDA’s cancer drug review office, in a statement. “The agency worked cooperatively with the companies to expedite the drug’s development, review and approval, reflecting the promise of the Breakthrough Therapy Designation program.”

The first approval is based on a study of 111 patients who got a daily dose of the oral pills. Researchers found that about two-thirds of patients (66 percent) had their tumors shrink after treatment, and an impressive 17 percent had their tumors completely disappear, according to a Pharmacyclics statement. The FDA said it still doesn’t know whether the drug extends survival time for patients with mantle cell lymphoma. It’s impossible for the pivotal study to answer that question, because it didn’t have a control group that randomly assigned patients to a placebo or some other standard therapy.

Like most cancer drugs, the new treatment comes with some pretty significant side effects. Almost one-tenth of patients dropped out of the pivotal study because of side effects, Pharmacyclics said in a statement. About 7 percent had moderate to severe pneumonia, while 5 percent reported to moderate to severe abdominal pain, diarrhea, fatigue, skin infections, and atrial fibrillation (irregular heartbeat). The drug’s prescribing information for physicians includes a warning about hemorrhages, kidney toxicity, infections, suppression of certain types of blood cells, and the possibility that patients could develop secondary malignancies.

Pharmacyclics CEO Bob Duggan said on a conference call with analysts today that the drug will cost $10,930 for mantle cell lymphoma patients who get a 30-day supply of the recommended dose. That pencils out to about $131,000 a year if a patient can take it on a chronic basis, making it one of the more expensive new cancer drugs on the U.S. market.

The drug is vitally important for Pharmacyclics, as it’s the first product it has developed for sale in the U.S., and the main driver of its $8.7 billion market valuation. Pharmacyclics struck a big partnership in 2011 with Johnson & Johnson, which brought $150 million in upfront cash to the smaller company, and a 50/50 split of the profits and losses.

The drug also has an interesting backstory behind it, as David Shaywitz detailed earlier this year in Forbes. Pharmacyclics picked up the drug cheap from Celera Genomics, the company which famously raced to sequence the first human genome, but never developed any drugs on its own. At least one oncologist told Shaywitz the new drug could be the “Gleevec of CLL” which is high praise, comparing the new product to Novartis’s imatinib (Gleevec). The Novartis drug turned chronic myeloid leukemia from a death sentence into something more like a chronic disease.

The FDA approval of the new treatment is also being watched closely by a number of contenders in the blood cancer field, especially on the eve of the big American Society of Hematology conference in New Orleans. The FDA recently approved Genentech’s obinutuzumab (Gazyva) for chronic lymphocytic leukemia, while Gilead Sciences (NASDAQ: GILD) and Infinity Pharmaceuticals (NASDAQ: INFI) are racing ahead with related drugs that work against the PI3 kinase molecular pathway.